Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Corporate Update (May 2026)
20 slides from Mirum Pharmaceuticals's corporate update, May 4, 2026
Slide 1: Mirum Pharmaceuticals VISTAS and AZURE-1 Phase 2b Topline Results
Mirum Pharmaceuticals VISTAS Phase 2b Topline Results AZURE-1 Phase 2b Topline Results M a y 4 , 2 0 2 6
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains “forward-looking” statements that are based on our management’s beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in
Slide 3: VISTAS Phase 2b Primary Sclerosing Cholangitis (PSC) Topline Results
VISTAS Phase 2b Primary Sclerosing Cholangitis (PSC) Topline Results 3
Slide 4: PSC: A Rare Immuno-Inflammatory Cholestatic Liver Disease
PSC: A Rare Immuno-Inflammatory Cholestatic Liver Disease Progressive Biliary Injury Drives Serious and Compounding No Approved Therapies Cholestasis and Liver Damage1 Disease Consequences1-3 Significant Need Pruritus and Fatigue Heavy symptom burden Progressive biliary strictures Elevated Laborator
Slide 5: PSC: Pruritus is Common and Often Moderate to Severe
PSC: Pruritus is Common and Often Moderate to Severe Pruritus is a Registrational Endpoint Pruritus Is a Significant Burden No Approved Therapies; Significant Opportunity in PSC 8 PSC US Patients with Pruritus Often Moderate to Severe Median worst itch score (0-10 NRS) from last itching episode1 ~30
Slide 6: Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus PSC patients with pruritus 28wk 2+ years VLX 20mg BID1 VLX Open Label Extension PBO Primary Endpoint Change in pruritus from baseline to 28wk2 Primary Cohort, n=111 (moderate to severe pruritus) Secondary Cohort, n=47 (mild prurit
Slide 7: VISTAS Phase 2b Baseline Demographics – Primary Cohort
VISTAS Phase 2b Baseline Demographics – Primary Cohort 20 mg Placebo (N=54) (N=57) Age at enrollment in years (SD) 45.2 (15.7) 44.3 (14.2) Sex (Female %) 25 (46.3) 35 (61.4) Adult ItchRO Score (SD) 6.3 (1.6) 6.1 (1.5) PSC Type (Large Duct %) 49 (90.7) 53 (93.0) sBA in mol/L (SD) 109.6 (137.5) 61.4
Slide 8: VISTAS Phase 2b Met Primary Endpoint
VISTAS Phase 2b Met Primary Endpoint RAPID, STATISTICALLY SIGNIFICANT REDUCTIONS IN PRURITUS Primary Cohort (moderate to severe pruritus) Volixibat Placebo 0 N=54 N=57 55.6% ≥2 point reduction in pruritus LS Mean change (SE) in the Adult -1.08 ItchRO** score from Baseline -1.64 -1 (0.241)* Serum Bil
Slide 9: VISTAS Phase 2b Safety Summary (Primary and Secondary Cohorts)
VISTAS Phase 2b Safety Summary (Primary and Secondary Cohorts) VLX 20 mg Placebo n (%) (N=77) (N=81) Participants with any treatment emergent adverse event (TEAE) 72 (93.5) 68 (84.0) Grade 3 or higher TEAEs 10 (13.0) 9 (11.1) Serious TEAEs 8 (10.4) 5 (6.2) TEAE that led to death 0 1 (1.2) TEAE that
Slide 10: TEAEs Occurring in ≥5% of Patients (Primary and Secondary Cohorts)
TEAEs Occurring in ≥5% of Patients (Primary and Secondary Cohorts) VLX 20 mg Placebo n (%) (N=77) (N=81) Diarrhea 31 (40.3) 7 (8.6) Abdominal Pain 14 (18.2) 8 (9.9) Nausea 10 (13.0) 3 (3.7) Vitamin D deficiency 8 (10.4) 4 (4.9) Abdominal pain upper 7 (9.1) 6 (7.4) ALT increased 7 (9.1) 3 (3.7) Upper
Slide 11: Volixibat Program Next Steps
Volixibat Program Next Steps FDA NDA pre-NDA Submission Meeting Topline Results Oral Late Breaker, Scheduled Planned Now Expected EASL International Summer 2026 H2 2026 Q1 2027 Liver Congress May 2026 11
Slide 12: AZURE Phase 2b Chronic Hepatitis Delta Virus (HDV) Topline Results
AZURE Phase 2b Chronic Hepatitis Delta Virus (HDV) Topline Results
Slide 13: HDV Is the Most Severe Form of Viral Hepatitis
HDV Is the Most Severe Form of Viral Hepatitis No Approved Therapies in the US Hepatitis Delta Virus >50% Liver-Related Death in 10 Years1 ~15,000~ US pts diagnosed, insured, under care4 5yrs Avg. Progression to Cirrhosis and Liver Failure2 ~40,000 Est. US Prevalence Requires Hepatitis B coinfection
Slide 14: Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV
Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV Brelovitug AZURE-1 Phase 2b/3 Study Design Fully human anti-HBsAg monoclonal antibody 24wk SC injection 1x Weekly or 1x Monthly 300mg 1x Weekly or 900mg 1x Monthly1 Extension (96wks) Delayed Tx→ 300mg 1x Weekly Delayed Tx Start Pri
Slide 15: AZURE-1 Phase 2b Baseline Demographics
AZURE-1 Phase 2b Baseline Demographics 300 mg QW 900 mg Q4W Delayed Treatment N=21 N=20 N=12 Age, years, mean (SD), range 45.8 (±7.0), 37 - 61 44.8 (±8.4), 26 - 58 49.0 (±9.9), 34 - 65 Men, n (%) 13 (62%) 15 (75%) 9 (75%) White, n (%) 19 (90%) 19 (95%) 11 (92%) Cirrhosis, n (%)1 10 (48%) 11 (55%) 7
Slide 16: AZURE-1 Phase 2b Week 24 Met Primary Endpoint
AZURE-1 Phase 2b Week 24 Met Primary Endpoint 300 mg QW 900 mg Q4W Delayed Treatment N=20 N=20 N=12 Virologic response 20/20 (100%) 15/20 (75%) 0/12 (0%) HDV RNA ≥2 log10 reduction or TND HDV RNA <LLOQ, TND 6/20 (30%) 1/20 (5%) 0/12 (0%) ALT normalization 9/20 (45%) 8/20 (40%) 1/12 (8%) Primary Endp
Slide 17: AZURE Phase 2b Safety
AZURE Phase 2b Safety 300 mg QW 900 mg Q4W Delayed Treatment Participants who experienced, n (%) N=21 N=20 N=12 AEs Any 11 (52) 10 (50) 3 (25) Related to treatment 7 (33) 7 (35) 0 Grade 3+ Any 1 (5)† 0 0 Related to treatment 0 0 0 Serious Any 0 1 (5)# 0 Related to treatment 0 0 0 AE Leading to Disco
Slide 18: Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions
Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions No ALT Limitation for Study Participation 24wk FDA Registration AZURE-1 n~200 2:2:1 300mg 1x Weekly vs 900mg 1x Monthly vs Delayed Tx Start Extension (96wks) Delayed Tx→ 300mg 1x Weekly Enabling Studies Primary Endpoint Week 24 Vir
Slide 19: Mirum Pharmaceuticals: Delivering High Impact Medicines for Rare Disease
Mirum Pharmaceuticals Delivering High Impact Medicines for Rare Disease
Slide 20: Well-Positioned to Execute on Our Planned Strategy
Well-Positioned to Execute on Our Planned Strategy 4 potentially registrational topline readouts expected in the next 12 months 2026 2027 2026 FY Guidance ✓ VISTAS (PSC) topline results in Q2 ❑ VANTAGE (PBC) topline results in Q1 ✓ AZURE-1 (HDV) Interim Analysis in Q2 ❑ Volixibat PSC Approval/Launch